Pharming Group Nv · EURONEXT:PHARM
Pharming Group Nv — Oppenheimer 35th Annual Healthcare Life Sciences Conference 2025
51 slides from Pharming Group Nv's conference presentation, February 2025
Slide 1: Pharming Group N.V. Oppenheimer 35th Annual Healthcare Life Sciences Conf.
Pharming Group N.V. Oppenheimer 35th Annual Healthcare Life Sciences Conf. February 2025 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 2: Forward-looking statements
Forward-looking statements This presentation may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management’s current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual resu
Slide 3: Building a leading global rare disease biopharma company
Building a leading global rare disease biopharma company Market RUCONEST® Global approvals and Ongoing pipeline development for acute HAE attacks in key commercialization of and management of rare markets – U.S. focus Joenja® (leniolisib) for APDS disease assets Positive cash flow from RUCONEST® Suc
Slide 4: RUCONEST (rhC1INH): trusted treatment cornerstone for HAE
RUCONEST® (rhC1INH): trusted treatment cornerstone for HAE The only recombinant treatment 2nd most prescribed product for that targets the root cause of HAE acute attacks by replacing missing or Typical patient: failed icatibant dysfunctional C1-INH (BK inh) and on prophy Tx (K inh) Well-tolerated a
Slide 5: Joenja (leniolisib) – strong execution and growth opportunity
Joenja® (leniolisib) – strong execution and growth opportunity Leniolisib for Primary Leniolisib (APDS) Immunodeficiencies Joenja® U.S. (APDS) (PIDs) Revenues: • Marketed (12+) • Found >870 • Phase II POC trial in • U.S. commercial sales patients globally PIDs with immune • Europe / RoW access progr
Slide 6: Pipeline – multiple commercial stage rare disease products
Pipeline – multiple commercial stage rare disease products Pending Abliva deal close 6
Slide 7: Joenja (leniolisib)
% Pharming’ Joenja’ (leniolisib)
Slide 8: U.S. launch of Joenja: first and only approved therapy for APDS
U.S. launch of Joenja®: first and only approved therapy for APDS, corrects the underlying immune defect Joenja® (leniolisib) is a prescription FDA approval (March 2023) based on medicine that is used to treat randomized pivotal study and OLE study activated phosphoinositide 3-kinase U.S. launch (Apr
Slide 9: Hiding in plain sight: Patient finding strategy
Hiding in plain sight: Patient finding strategy Medical education to raise awareness of APDS Genetic testing VUS resolution and share leniolisib data Conferences and congresses Sponsored, no-cost Validation studies with various Abstracts testing program laboratories to confirm which Assistance from
Slide 10: Joenja geographic / pediatric / indication expansion
Joenja® ‒ geographic / pediatric / indication expansion Europe – CHMP review extended to January 2026 Israel marketing authorization Single outstanding CMC request received April 30, 2024 Positive clinical benefit and safety concluded U.K. marketing authorization CAN, AUS submissions received Septem
Slide 11: Leniolisib for PIDs with immune dysregulation
Leniolisib for PIDs with immune dysregulation Primary Immunodeficiencies (PIDs) are a broad group of disorders with key potential features: • Genetic basis • Immune dysfunction → increased risk of infection • Immune dysregulation → lymphoproliferation and autoimmunity PIDs • High morbidity and morta
Slide 12: Proposed Abliva AB Acquisition (KL1333)
Proposed Abliva AB Acquisition (KL1333)
Slide 13: Acquisition terms and financial details
Acquisition terms and financial details Acquisition Terms Financial Details Timing Acquisition through a public Acquisition of shares with Offer was declared unconditional tender offer under Swedish available cash and completed on 7 February 2025 Takeover Act and Nasdaq with 87.7% of shares tendered
Slide 14: Attractive rare disease opportunity with blockbuster potential
Attractive rare disease opportunity with blockbuster potential Mitochondria are the “powerhouses” of cells • Producing chemical energy to power the human body, in particular muscle cells Primary mitochondrial diseases are rare disorders affecting the mitochondria’s ability to generate energy • Patie
Slide 15: Pivotal study design based on regulatory and patient advocacy input
Pivotal study design based on regulatory and patient advocacy input Study Schematic Regulatory Feedback Study Design Screening Both FDA and EMA accepted Methodology study as registrational • Randomized, double-blind, parallel-group, placebo- FDA said achieving one of the controlled pivotal study 3:2
Slide 16: FALCON study – positive interim analysis
FALCON study – positive interim analysis Pivotal FALCON Study Interim Futility Analysis: Positive outcome achieved, with both primary endpoints having WAVE 1 – Fully enrolled passed futility 40 patients recruited across six countries (U.S., UK, France, Spain, Belgium, Denmark) Promising differences
Slide 17: First-in-disease rare disease therapy with blockbuster potential
First-in-disease rare disease therapy with blockbuster potential Builds on Pharming’s existing rare disease expertise and infrastructure KL1333 positioned to become first standard of care in mtDNA mitochondrial disease Large commercial opportunity with >30,000 mtDNA diagnosed patients in US, EU4 and
Slide 18: Financials and Outlook
% Pharming’ Financials and Outlook
Slide 19: Financial highlights: 3Q 2024 vs 3Q 2023
Financial highlights: 3Q 2024 vs 3Q 2023 Revenues Gross profit OpEx development RUCONEST® Joenja® R&D G&A M&S Amounts in US$ million Amounts in US$ million Amounts in US$ million 74.8 68.0 64.7 66.7 11.2 56.8 6.5 58.4 28.7 25.1 60.2 63.6 10.9 15.3 20.8 20.7 3Q 2023 3Q 2024 3Q 2023 3Q 2024 3Q 2023 3Q
Slide 20: Pharming 2024 Outlook
Pharming 2024 Outlook Total revenues between US$280 and US$295 million (14% to 20% growth). Joenja® (leniolisib) U.S.: Continued progress finding additional APDS patients, supported by family testing and VUS validation efforts, and subsequently converting patients to paid therapy. Leniolisib ex-U.S.
Slide 21: www.pharming.com
www.pharming.com NASDAQ: PHAR | EURONEXT Amsterdam: PHARM
Slide 22: Appendix
Pharming Group N.V. Appendix
Slide 23: C1-INH targets the root cause of HAE
C1-INH targets the root cause of HAE There are 3 known cascades that can lead to an HAE attack Complement System1-6 Contact Activation System1,7-10 Fibrinolysis System8,14 Classical Lectin C1-INH therapy regulates Trigger(s)12 Pathway Pathway FXII multiple pathways11 Immune Complexes • Trauma Plasmi
Slide 24: C1-INH targets the root cause of HAE: addresses unmet patient needs
C1-INH targets the root cause of HAE Addresses patient needs unmet by other therapies TARGET BRAND GENERIC NAME STATUS TYPE NAME C1 Inhibitor Overview of Marketed and In-Development Therapies and Their Ruconest C1 esterase inhibitor (recombinant) Marketed OD Targets Within the Three Known Cascades L
Slide 25: APDS is a rare primary immunodeficiency: PI3Kδ hyperactivity
APDS is a rare, primary immunodeficiency (PID) Genetic defect leads to PI3Kδ hyperactivity Hyperactive PI3Kδ results in dysregulated Immune imbalance leads to diverse signs B and T cell development1-3 and symptoms1,4-6 Severe, recurrent, persistent infections The PI3Kδ enzyme • Sinopulmonary p110δ •
Slide 26: APDS is a rare, primary immunodeficiency first characterized in 2013
APDS is a rare, primary immunodeficiency (PID) first characterized in 2013 Activated phosphoinositide 3- kinase delta (PI3Kδ) syndrome A genetic test can provide a definitive (APDS) diagnosis of APDS Global prevalence estimated at 1.5 patients per million population* To date, Pharming has identified
Slide 27: APDS can impact many facets of life
APDS can impact many facets of life Physical1,2 Frequent infections Social3,4 Swollen glands Missing school, work, or Shortness of breath daily activities Coughing/wheezing Chest or joint pain Fatigue Treatment Burden1-4 Inability to exercise Frequent hospitalizations Hearing loss Surgeries Diarrhea
Slide 28: Heterogeneous, evolving symptomology can lead to missed diagnoses
Heterogeneous, evolving symptomology can often lead to missed diagnoses Timeline of the most common pathologies* seen in APDS1-4 Median age at diagnosis: 12 years (7-year median diagnosis delay) <1 year 3 years 5 years 10.5 years 11.2 years† 18 years (range, 1 month-10 years) (range, 1-6 years) (ran
Slide 29: Management for APDS prior to Joenja
Management for APDS1,2 prior to Joenja® Immune Deficiency Immune Dysregulation • Antimicrobial prophylaxis • Corticosteroids • Immunoglobulin • Other immunosuppressants replacement therapy • mTOR inhibitors None of these therapies are FDA- approved for APDS treatment Hematopoietic stem cell transpla
Slide 30: Joenja clinical trial designs
Joenja® clinical trial designs Pivotal Trial - Pivotal Trial - Part 2: Open-label Part 1: Efficacy extension Dose- & Safety study4,5 finding1,2 Evaluation3 Nonrandomized, open-label, Randomized, triple-blinded, Nonrandomized, open-label, dose-escalating placebo-controlled long-term study 31 patients
Slide 31: Joenja addresses the underlying cause of APDS – Phase 3 co-primary endpoints
Joenja® addresses the underlying cause of APDS to help restore immune balance – Phase 3 co-primary endpoints At 12 weeks Joenja® decreased lymphadenopathy and increased naïve B cells Change from baseline in index nodes* Change from baseline in naïve B cells† Log10-transformed SPD of index lesions Jo
Slide 32: Joenja significantly reduced splenomegaly
Joenja® significantly reduced splenomegaly Secondary endpoint: Significant reductions in at week 12 spleen size by 2D and 3D analysis compared to placebo 27% reduction in 3D spleen volume* • The adjusted mean difference in bidimensional spleen size between Joenja® (n=19) and placebo (n=9) was -13.5
Slide 33: Joenja reduced IgM levels
An exploratory end point showed Joenja® reduced IgM levels Mean serum IgM rapidly reduced to within normal limits • In the Joenja® arm, IgM was 600 elevated above normal limits in 6 patients at baseline, and by week 12 was reduced in all, with 50% returning to IgM, mg/dL 400 within normal limits • I
Slide 34: Joenja safety profile
Joenja® safety profile Phase 3 Trial1,2 Open-label Extension Study3 Data cutoff for interim analysis: December 13, 2021 Adverse reactions reported by ≥2 patients treated with Joenja and more frequently than placebo Joenja (n=21) Placebo (n=10) • 32/37 patients Most common AEs n n (%) n (%) reported
Slide 35: Open-label extension interim analysis of infections and IRT reduction
Open-label extension interim analysis of days spent with infections and IRT reduction Annual Rate of Infection Days* Physician-reported IRT Reduction‡ 45 40 42.4 Of 27 patients receiving IRT: Days with infection per patient (m/N) Baseline† Baseline† 35 39.0 38.5 30 25 Baseline† 26.7 20 Baseline† 22.
Slide 36: VUS by the numbers
VUS by the numbers VUSs frustrate patients and doctors, limiting diagnosis of genetic diseases such as APDS Pharming is aware of ~1,200 US patients harboring PIK3CD/R1 VUSs • This figure will continue to grow over time • VUS are identified at ~4x the rate of likely pathogenic/pathogenic (LP/P) varia
Slide 37: Immune dysregulation in PIDs can occur via altered PI3Kδ signaling
Given importance of PI3Kδ in B & T cells, immune dysregulation in PIDs can occur via alterations in PI3Kδ signaling Altered PI3Kδ signaling can occur in multiple Clinical manifestations, disease onset PID genetic disorders beyond APDS and severity similar to APDS 5-10 (ex: ALPS-FAS, CTLA4, NFKB1, PT
Slide 38: PIDs linked to PI3Kδ signaling – Phase II study design
PIDs linked to PI3Kδ signaling – Phase II study design Phase II proof of concept clinical trial – single arm, open- label, dose range-finding study (N=12) 10mg 30mg 70mg • Patients with PIDs linked to PI3Kδ signaling, e.g. Lead Investigator: Gulbu Uzel, M.D., Senior ALPS-FAS1, CTLA4 haploinsufficien
Slide 39: Epidemiology of PIDs linked to PI3K signaling
Epidemiology of PIDs linked to PI3K signaling Current/Future9 % Targeted for Targeted Genetic PID Prevalence References Prevalence Estimate Leniolisib Population Type (per million) Treatment (per million) ESID registry (236 patients): 6 /mill.1 ALPS-FAS 5/10 30%3 3 PID KOLs – 4 /mill2 ESID registry
Slide 40: RUCONEST and Joenja driving revenue growth
RUCONEST® and Joenja® driving revenue growth 300 Pharming revenues FY 2016 – FY 2023 250 245.3 18.2 Total revenues in $* millions 200 150 227.1 100 212.2 205.6 189.4 198.9 159.6 50 101.3 17.5 0 2016 2017 2018 2019 2020 2021 2022 2023 RUCONEST® Joenja® • • From FY 2016 – FY 2020 Pharming Group report
Slide 41: OpEx development – 2024 quarterly
OpEx development – 2024 quarterly Operating Costs 1Q24 – 3Q24 (R&D, G&A, M&S) 70.1 63.8 64.7 Amounts in US$ million 1Q24 2Q24 3Q24 41
Slide 42: 2024 Financial guidance
2024 Financial guidance FY 2024 Revenue Guidance % Growth vs. FY 2023 Total Revenues US$280 - 295 million 14-20% Joenja® significant driver of revenue growth, continued RUCONEST® growth Joenja® revenue assumptions: • Continued growth in patients on paid therapy • Continued high (monthly) adherence (
Slide 43: Appendix: Proposed Abliva AB Acquisition (KL1333)
Pharming Group N.V. Appendix Proposed Abliva AB Acquisition (KL1333)
Slide 44: Dysfunctional mitochondria produce less ATP
Dysfunctional mitochondria produce less ATP Primary Mitochondrial Disease (PMD) Mitochondria, often described as the “powerhouses” Dysfunctional Mitochondria of cells, are crucial for energy production Mutation Impaired ETC protein expression Mitochondrial diseases are a group of genetic disorders c
Slide 45: Heavy patient burden with no approved therapies
Heavy patient burden with no approved therapies Presentation and Diagnosis Patients present to their primary care doctor and then often get referred to a neurologist for musculoskeletal issues Either the neurologist or a referral to a metabolic geneticist will result in a diagnosis Many patients are
Slide 46: KL1333 corrects the underlying pathophysiology
KL1333 corrects the underlying pathophysiology KL1333 normalizes conversation of NADH to NAD+ via NQO1 Normalizes the NAD+/NADH Ratio Restored Energy Metabolism Electron Transport Chain Complex III Complex IV Mitochondrial Membrane Q Synthase ATP Complex I Complex II X NADH NAD+ ADP + Pi ATP Restore
Slide 47: KL1333: First-in-disease small molecule with unique MOA
KL1333: First-in-disease small molecule with unique MOA Attributes Outcomes Directly increases the NAD+/NADH ratio Improved energy regulation and ETC via NQO1 function Unique MoA works upstream from all competing MoA in PMD Stimulation of mitochondria biogenesis Oral, small molecule, BID dosing Fati
Slide 48: Phase 1b demonstrated significant activity vs. placebo
Phase 1b demonstrated significant activity vs. placebo The placebo-controlled Phase 1b study demonstrated that KL1333 reduced patients’ fatigue and myopathy after only 10 days, 50 mg/day Fatigue Reduction Muscle Function Improvement Changes from baseline to day 10 Changes from baseline to day 10 KL1
Slide 49: Significant revenue opportunity for KL1333
Significant revenue opportunity for KL1333 PMD Prevalence 1 per 4,3001 US 78,200; EU4 and UK 76,400 Diagnosed mtDNA 9.6 per 100,0002 US 32,300; EU4 and UK 31,600 FALCON Trial Inclusion Criteria* 51% US 16,500; EU4 and UK 16,100 >30,000 diagnosed mtDNA mitochondrial disease patients addressable in th
Slide 50: Majority of patients diagnosed and treated in US Centers of Excellence
Majority of patients diagnosed and treated in US Centers of Excellence or academic institutions Mitochondrial Care Network – Centers: www.mitonetwork.org/centers 50
Slide 51: Other programs focus on different patient population or failed with different MOA
Other programs focus on different patient population or failed with different MOA Previous programs failed due to old mechanisms of action or evaluating the wrong endpoints Asset Type MOA / ROA Stage Patient Group Comments mtDNA mutations (e.g., NAD+/NADH ▪ Ongoing potentially registrational phase 2
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